Product Description
Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 (CLN2), a paediatric neurodegenerative disease caused by a deficiency in TPP1. (Sourced from: https://pubmed.ncbi.nlm.nih.gov/28589525/)
Mechanisms of Action: TPP1 Modulator
Novel Mechanism: No
Modality: Coagulation Factor
Route of Administration: N/A
FDA Designation: None *
Approval Status: Approved
Approved Countries: Australia | Austria | Belgium | Brazil | Canada | Croatia | Czech | Denmark | Estonia | European Medicines Agency | Finland | Germany | Greece | Hungary | Iceland | Ireland | Israel | Italy | Japan | Jordan | Korea | Latvia | Lithuania | Netherlands | Poland | Portugal | Romania | Slovakia | Slovenia | Sweden | Taiwan | Ukraine | United Kingdom | United States
Approved Indications: CLN2 | Neuronal Ceroid-Lipofuscinosis
Known Adverse Events: Bradycardia | Hypotension | Seizures | Headache
Company: Biomarin
Company Location: NOVATO CA 94949
Company CEO: Jean-Jacques Bienaimé
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Germany, Italy, United Kingdom, United States
Active Clinical Trial Count: 1
Highest Development Phases
Phase 2: CLN2|Neuronal Ceroid-Lipofuscinosis
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
---|---|---|---|---|---|
NCT02678689 | P2 |
Completed |
CLN2|Neuronal Ceroid-Lipofuscinosis |
2022-04-20 |
32% |